Literature DB >> 29074441

Marketing authorisation of orphan medicines in Europe from 2000 to 2013.

Matthias P Hofer1, Hanna Hedman1, Maria Mavris1, Franz Koenig2, Thorsten Vetter1, Martin Posch2, Spiros Vamvakas1, Jan Regnstrom1, Stiina Aarum3.   

Abstract

An analysis was performed on a data set of 157 orphan designated medicines with an outcome for marketing authorisation application (MAA) between 2000 and 2013. The intention was to understand the factors associated with marketing authorisation success, the challenges developers face regarding orphan medicine development, and how scientific advice (SA) is used during development. The results demonstrated that orphan medicines have a lower success rate compared with non-orphan medicines and that determinants for marketing authorisation success were company size and compliance with SA. Compliance with SA could help orphan medicine developers overcome clinical development challenges.
Copyright © 2017 The Authors. Published by Elsevier Ltd.. All rights reserved.

Mesh：

Year:  2017        PMID： 29074441     DOI： 10.1016/j.drudis.2017.10.012

Source DB:  PubMed          Journal:  Drug Discov Today        ISSN： 1359-6446            Impact factor:   7.851


  8 in total

Review 1.  [Orphan drugs from the perspective of the Drug Commission of the German Medical Association : Opportunities and challenges].

Authors:  Wolf-Dieter Ludwig
Journal:  Internist (Berl)       Date:  2019-04       Impact factor: 0.743

Review 2.  [Evidence-based health care with pharmaceuticals for rare diseases: the role of digitalisation].

Authors:  Frauke Naumann-Winter; Thomas Kaiser; Antje Behring
Journal:  Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz       Date:  2022-10-20       Impact factor: 1.595

3.  Generation of therapeutic antisera for emerging viral infections.

Authors:  Rebecca Schmidt; Lea C Beltzig; Bevan Sawatsky; Olga Dolnik; Erik Dietzel; Verena Krähling; Asisa Volz; Gerd Sutter; Stephan Becker; Veronika von Messling
Journal:  NPJ Vaccines       Date:  2018-10-05       Impact factor: 7.344

4.  Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review.

Authors:  Sarah Goring; Aliki Taylor; Kerstin Müller; Tina Jun Jian Li; Ellen E Korol; Adrian R Levy; Nick Freemantle
Journal:  BMJ Open       Date:  2019-02-27       Impact factor: 2.692

5.  Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval.

Authors:  Kenji Harada; Kazuki Toriyabe; Shunsuke Ono
Journal:  J Clin Pharmacol       Date:  2019-07-31       Impact factor: 3.126

6.  Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation.

Authors:  Jorn Mulder; Tobias van Rossum; Segundo Mariz; Armando Magrelli; Anthonius de Boer; Anna M G Pasmooij; Violeta Stoyanova-Beninska
Journal:  Front Oncol       Date:  2021-12-20       Impact factor: 6.244

Review 7.  Lessons learned from IDeAl - 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials.

Authors:  Ralf-Dieter Hilgers; Malgorzata Bogdan; Carl-Fredrik Burman; Holger Dette; Mats Karlsson; Franz König; Christoph Male; France Mentré; Geert Molenberghs; Stephen Senn
Journal:  Orphanet J Rare Dis       Date:  2018-05-11       Impact factor: 4.123

8.  Pricing and Reimbursement Pathways of New Orphan Drugs in South Korea: A Longitudinal Comparison.

Authors:  Jong Hyuk Lee
Journal:  Healthcare (Basel)       Date:  2021-03-08
  8 in total

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